The company will use the proceeds from the Series C investment round to support the advancement of PTX-022 (QTORIN 3.9 percent rapamycin anhydrous gel) for adults with Pachyonychia Congenita (PC), a rare, chronically debilitating and lifelong genetic disease
Fremont, CA: Biopharmaceutical company focusing on rare diseases, Palvella Therapeutics, secured USD 45 million in an oversubscribed Series C funding round participated by CAM Capital, Samsara BioCapital, BVF Partners L.P., Adams Street Partners, Opaleye Management, Ligand Pharmaceuticals (Nasdaq: LGND), Agent Capital, BioAdvance and Nolan Capital, the investment fund of former AveXis CEO Sean Nolan. As part of the investment round, Scott Morenstein, Managing Director of CAM Capital, and Cory Freedland, Principal of Samsara BioCapital, will join Palvella's Board of Directors.
"Palvella was founded on fundamental beliefs that every individual with a rare disease deserves a treatment and that significant value creation occurs upon the introduction of the first approved therapy for a serious rare genetic disease," stated Wes Kaupinen, President and Chief Executive Officer of Palvella. "The capital invested from this highly regarded syndicate of the public market and venture investors strengthens our team's unwavering commitment to develop and commercialize targeted therapies to individuals suffering from serious, life-altering rare genetic diseases such as pachyonychia Congenita (PC) and Gorlin syndrome."
The company will use the proceeds from the Series C investment round to support the advancement of PTX-022 (QTORIN 3.9 percent rapamycin anhydrous gel) for adults with Pachyonychia Congenita (PC), a rare, chronically debilitating and lifelong genetic disease. Individuals suffering from PC experience extreme pain and difficulty with ambulation, frequently necessitating the use of either ambulatory aids or alternative forms of mobility such as crawling on hands and knees. To date, there is no approved treatment for PC. Estimates suggest that atleast 9,000 individuals in the U.S. suffer from PC.
Palvella completed enrollment in March 2020 of the Phase 2/3 pivotal VALO Study for PTX-022 in partnership with Pachyonychia Congenita Project. PTX-022 is a novel formulation of rapamycin, which was shown by geneticist Dr. Roger Kaspar to have a direct mechanism of action on mutant keratin genes, which are the root cause in PC. PTX-022 leverages Palvella's QTORIN™ technology to enable localized distribution of rapamycin into the suprabasal keratinocytes, which express the mutant keratin genes causing a primary defect in PC. Palvella expects top-line results from VALO to be available in the fourth quarter of 2020.